Association of long-term corticosteroid therapy with left ventricular systolic dysfunction in adults with duchenne muscular dystrophy: a single centre cross-sectional study
L V Sese, M C GuillermoAbstract
Background / Introduction
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder characterised by progressive skeletal and cardiac muscle degeneration, with dilated cardiomyopathy representing the main cause of adult cardiac mortality. Although corticosteroids are established disease-modifying therapy in childhood and adolescence, their long-term cardiac impact in adults remains unclear. Identifying modifiable factors associated with cardiomyopathy is important as advances in multidisciplinary care have increased life expectancy in DMD. As a result, this growing population is experiencing an increasing burden of cardiac-related morbidity and mortality, with progressive left ventricular systolic dysfunction emerging as an important prognostic factor.
Purpose
To evaluate whether ongoing corticosteroid therapy is associated with reduced left ventricular ejection fraction (LVEF ≤40%) in adults with genetically confirmed DMD.
Methods
A cross-sectional study was performed. Adults with DMD under follow-up at an accredited Duchenne neuromuscular centre in the United Kingdom as of January 2025 were reviewed. Current corticosteroid use was recorded (both intermittent and continuous). LVEF was obtained from the most recent echocardiogram and classified as ≤40% or >40%. Binary logistic regression was used to evaluate the association between corticosteroid use and LVEF, adjusting for age, body mass index, genotype, comorbidities such as atrial fibrillation, hypertension, Type 2 diabetes, chronic kidney disease, non-invasive ventilation use, and cystatin C levels. Heart failure medications were excluded from the model to avoid confounding by indication.
Results
Among 123 adults with DMD (mean age 23.5 ± 4.2 years), 84 (68%) were on steroids (66 on prednisolone and 15 on deflazacort). Among those on steroids, 35 were on a daily regimen while 49 were on an intermittent/10 days on then off regimen. With regards to the outcome, 49 (40%) had LVEF ≤40%. Following logistic regression, ongoing corticosteroid therapy was independently associated with significantly lower odds of reduced systolic function (adjusted OR 0.36; 95% CI 0.13–0.99; p=0.049). Other covariates in the model did not show statistically significant associations with LVEF status.
Conclusions
In this adult DMD cohort, long-term corticosteroid therapy was associated with markedly lower odds of having LVEF ≤40%. These results support the possibility that continuation of steroid therapy into adulthood may have cardioprotective effects in addition to its known benefits on skeletal muscle stability. Prospective longitudinal studies are required to confirm causality and determine optimal long-term steroid strategies for preserving cardiac function in adult Duchenne patients.